Last fall, the RNA medicine startup Orbital Therapeutics launched in Cambridge, MA. Their mission, they say, is focused on enhancing global health by unleashing the full potential of RNA medicines to treat human disease. This week, they announced the successful closing of $270 million Series A financing.

Orbital Therapeutics aims to expand the applicability of RNA-based medicines across a range of human diseases. This includes next-generation vaccines, immunomodulation, and protein replacement. The company is building an RNA platform that integrates both established and emerging RNA technologies and delivery mechanisms.

“In recent years, we have only just started to see the expansive potential RNA-based therapeutics may offer to the future of medicine and how we treat disease,” said Giuseppe Ciaramella, PhD, Orbital co-founder and CEO. “Since our founding, we have been diligently focused on advancing our platform of RNA technologies to elucidate the most promising opportunities with our initial programs so we may reach patients as efficiently as possible. We are thrilled to have the backing of such an exceptional group of investors, whose support further enables the growth of our organization and the meaningful expansion of our platform and portfolio.”

The Series A raise was led by ARCH Venture Partners, with participation from initial investors a16z Bio + Health and Newpath Partners, and new investors Abu Dhabi Growth Fund (ADG), Redmile Group, Exor N.V., Invus, Moore Strategic Ventures, iGlobe Platinum Fund Group, Casdin Capital, Agent Capital, Alexandria Venture Investments, Rellim Capital Management, Heritage Medical Systems, and other undisclosed investors.

In other RNA news, the London-based MiNA Therapeutics announced that they are entering into a research collaboration with BioMarin Pharmaceutical to advance their small activating RNA (RNAa) platform in rare genetic diseases. The collaboration will allow for the discovery, and potential development and commercialization, of RNAa therapeutic candidates targeting a number of rare genetic diseases.

MiNA’s RNAa platform has been clinically validated in more than 120 patients to date. The universally designed platform has the potential to address any gene given its endogenous mechanism of action that ultimately addresses the root cause of the defective gene and enables the body to self-correct. Furthermore, working at the gene level, RNAa medicines are able to restore a cell’s own biology. This holds the potential to treat currently “undruggable” diseases.

Under the agreement, BioMarin will employ MiNA Therapeutics’ proprietary RNAa algorithm and technology platform to identify and characterize RNAa molecules targeting a number of genetic diseases for which there are currently no or minimal therapeutic options. The agreement does not cover oncology, or other therapeutic areas outside of genetic disease. BioMarin may then elect to license the assets fully for worldwide development and commercialization.

As part of the deal, MiNA will receive an upfront payment, and future royalties will be subject to BioMarin exercising options from the deal. Financial terms of the agreement were not disclosed.

“Activating RNA therapeutics have the potential to change the way we treat certain genetic diseases, particularly those characterized by the limited production of key proteins,” said Kevin Eggan, PhD, senior vice president and CSO of BioMarin.

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